UA News

The work of Cancer Prevention Pharmaceuticals may one day help prevent colon cancer in those at high risk for the disease.

The Arizona Board of Regents recently gave its approval for a University of Arizona researcher to take on part ownership of the new Tucson company, Cancer Prevention Pharmaceuticals, known as CPP. CPP's work may one day help prevent colon cancer in those at high risk for the disease.

Eugene Gerner, a UA professor of cell biology and anatomy, will run CPP with his colleague Frank L. Meyskens Jr., a professor of medicine and director of the Chao Family Comprehensive Cancer Center at the University of California, Irvine.

Gerner is a member of UA's BIO5 Institute and the Arizona Cancer Center, where he is the lead investigator on the National Cancer Institute-funded Specialized Program of Research Excellence, or SPORE, in gastrointestinal cancers grant.

CPP currently is conducting FDA-approval trials on Eflornithine, which in combination with an existing drug, has been shown in earlier clinical trials to reduce the recurrence of colon polyps by 70 percent, and has reduced the recurrence of the type of polyps most strongly associated with cancer by 92 percent. Colon polyps of all types are possible precursors to colon cancer. Gerner, who has been studying Eflornithine for more than two decades, is hopeful that the drug could be available in as little as three years.

While there are many cancer treatment drugs available, drugs for preventing the disease are still rare. "What we're trying to do is pretty unique," Gerner said. "Our mission is to bring drug-based prevention into the clinical management of patients at high risk for cancer."

That uniqueness initially presented a challenge. Gerner and Meyskens found that drug companies were unwilling to take on the liability and uncertain profits associated with a prevention-based treatment. Instead, Gerner and Meyskens formed CPP to assure a pathway for Eflornithine from the laboratory to the clinic.

Eflornithine disrupts the body's synthesis of polyamines, compounds associated with abnormal cell growth and cancer in adults. In combination with Sulindac, an already available non-steroidal anti-inflammatory drug, the paired drugs disrupt polyamine production and inflammation also associated with cancer.

Earlier Eflornithine clinical trials chiefly focused on safety and on finding the lowest possible dose that would still be effective. Gerner said reducing side effects was critical for a drug meant to be taken not only by cancer patients, but also by currently healthy individuals at risk for cancer. Ultimately the researchers found a dose so low it had no more side effects than a placebo containing no medication, but still was effective.

"We're hopeful that the FDA approval trials will lead to physicians being able to prescribe and patients being able to obtain Eflornithine," Gerner said. CPP also is working on ways to target patients who are at the greatest risk of colon cancer, including those who've had colon polyps in the past and those with strong family histories, since they stand to benefit most from the drug.

Gerner and Meyskens said Eflornithine may one day help prevent other cancers as well. Preliminary studies indicate that it is effective in men with a high risk of prostate cancer. Studies are currently underway among women who are at risk for breast cancer.

The multi-million dollar SPORE grant is funding a portion of the current trials, and additional grants are planned by Arizona Cancer Center investigators based on availability of Eflornithine, all of which bring further economic benefits to the state. The UA and the University of California hold the patent on the use of Eflornithine in cancer prevention and would receive a share of the profits from its prescription to patients.

Gerner says the new drug could lower cancer mortality rates significantly as well as the health costs associated with cancer treatment. "Our company is working to treat patients with relatively inexpensive drugs that have both low risks and very positive benefits," he said.